
Case Report
Longitudinal Data in Patients with Niemann-Pick Type C Disease Under Combined High Intrathecal and Low Intravenous Dose of 2-hydroxylpropyl-β-cyclodextrin
by Evangelia Bountouvi, MD, PhD; Melpomeni Giorgi, MD; Anna Papadopoulou,
by Evangelia Bountouvi, MD, PhD; Melpomeni Giorgi, MD; Anna Papadopoulou,
by Philip B Cawkwell, MD; Ivan Daniel Mayor, BA; and
by Atmaram Yarlagadda, MD; Samuel L. Preston, DO; Rachel P.
by Michele Meglio, PhD Student; Enrica Olivola, MD, PhD; Marco
[protected]by Aksha M. Memon, MD, MPH Dr. Memon is an
by Takahiko Nagamine, MD, PhD Dr. Nagamine is with the
Dear Editor: We read the excellent article by Diaz-Abad et
by Yousra BENMAKHLOUF, PhD; Zeineb ZIAN, PhD; Kaoutar BEN MAKHLOUF;
by Rocco Salvatore Calabrò, MD, PhD; Claudia Müller-Eising, Dr. jur.;
by Sushma Dahal, MPH; Meghnath Dhimal, PhD; Sagun Ballav Pant,